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Long-term safety and effectiveness of canakinumab therapy in patients with cryopyrin-associated periodic syndrome: results from the β-Confident Registry
- Source :
- RMD Open, Vol 7, Iss 2 (2021), RMD Open, RMD open, 7(2):e001663. BMJ Publishing Group
- Publication Year :
- 2021
- Publisher :
- BMJ, 2021.
-
Abstract
- ObjectiveTo report the long-term safety and effectiveness of canakinumab, a fully human anti-interleukin 1β monoclonal antibody, in patients with cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset multisystem inflammatory disease (NOMID), in a real-world setting.MethodsFrom December 2009 to December 2015, the β-Confident Registry prospectively enrolled patients with CAPS and non-CAPS conditions who received canakinumab per routine care and were prospectively followed for up to 6 years. The registry protocol did not mandate specific visits or procedures; however, all observed adverse events (AEs) and serious adverse events (SAEs) had to be recorded. Canakinumab effectiveness was evaluated by Physician’s Global Assessment (PGA).ResultsOf 288 patients enrolled, 3 were excluded due to missing informed consent. Among the remaining 285 patients, 243 (85.3%) were patients with CAPS and 42 (14.7%) had atypical CAPS (6.3%) or other conditions (8.4%). The median age was 26.6 years. Based on PGA, 58 of 123 (47.2%) patients with CAPS had no disease activity at 48 months, and 65 of 123 (52.8%) experienced mild/moderate disease activity at 48 months. Among CAPS phenotypes, AE incidence rates per 100 patient-years were lowest for FCAS (73.1; 95% CI 60.3 to 87.8) compared with those with MWS (105.0; 95% CI 97.2 to 113.2) or NOMID (104.6; 95% CI 86.6 to 125.2). One hundred twenty-eight SAEs were reported in 68 patients with CAPS (incidence rate/100 patient-years, 14.0; 95% CI 11.6 to 16.6). One death (metastatic rectal adenocarcinoma in a patient with MWS) was reported.ConclusionsThe response to canakinumab was sustained for up to 6 years. Canakinumab demonstrated a favourable safety profile over long-term treatment in patients with CAPS.Trial registration numberNCT01213641.
- Subjects :
- Adult
medicine.medical_specialty
cryopyrin-associated periodic syndromes
Immunology
Disease
Antibodies, Monoclonal, Humanized
03 medical and health sciences
0302 clinical medicine
Rheumatology
Familial Cold Autoinflammatory Syndrome
Informed consent
Internal medicine
medicine
Rectal Adenocarcinoma
Humans
Immunology and Allergy
In patient
Registries
030212 general & internal medicine
Adverse effect
030203 arthritis & rheumatology
business.industry
Antibodies, Monoclonal
Cryopyrin-associated periodic syndrome
medicine.disease
Autoinflammatory Disorders
Canakinumab
biological therapy
inflammation
Medicine
business
medicine.drug
Subjects
Details
- ISSN :
- 20565933
- Volume :
- 7
- Database :
- OpenAIRE
- Journal :
- RMD Open
- Accession number :
- edsair.doi.dedup.....1e6d45be128d162beb57777920ddeb35